Patisiran

DRUG8 trials

Sponsors

Alnylam Pharmaceuticals, Austin Neuromuscular Center

Conditions

ATTR Amyloidosis With CardiomyopathyAmyloidosisAmyloidosis, FamilialAmyloidosis, HereditaryHereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathiesPolyneuropathyTTR-mediated Amyloidosis

Early Phase 1

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

CompletedNCT05023889

Austin Neuromuscular CenterPolyneuropathies, Transthyretin Amyloidosis, Wild Type ATTR Amyloidosis +2

Start: 2022-08-03End: 2026-04-13Updated: 2026-05-22

Phase 2

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

CompletedNCT01617967

Alnylam PharmaceuticalsTTR-mediated Amyloidosis

Start: 2012-05-31End: 2014-01-31Updated: 2024-04-19

Phase 3

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

CompletedNCT02510261

Alnylam PharmaceuticalsAmyloidosis

Start: 2015-07-16End: 2022-11-23Updated: 2023-12-06

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

CompletedNCT03759379

Alnylam PharmaceuticalsAmyloidosis, Hereditary, Transthyretin Amyloidosis

Start: 2019-02-14End: 2025-11-05Updated: 2026-01-12

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

CompletedNCT03862807

Alnylam PharmaceuticalsAmyloidosis, Familial, Transthyretin Amyloidosis

Start: 2019-03-27End: 2020-10-20Updated: 2024-04-22

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

CompletedNCT03997383

Alnylam PharmaceuticalsTransthyretin Amyloidosis (ATTR) With Cardiomyopathy

Start: 2019-09-04End: 2025-12-24Updated: 2026-04-20

Unknown Phase

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

CompletedNCT04201418

Alnylam PharmaceuticalsHereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy

Start: 2019-12-18End: 2022-05-24Updated: 2022-06-06

Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

Alnylam PharmaceuticalsATTR Amyloidosis With Cardiomyopathy, Transthyretin-mediated Amyloidosis With Cardiomyopathy

Updated: 2024-02-16

Related Papers

Long-term efficacy and safety of vutrisiran in hereditary transthyretin amyloidosis with polyneuropathy: final analysis of the HELIOS-A randomized treatment extension

Figshare2026-07-27

Long-term efficacy and safety of vutrisiran in hereditary transthyretin amyloidosis with polyneuropathy: final analysis of the HELIOS-A randomized treatment extension

Figshare2026-06-15

Long-term efficacy and safety of vutrisiran in hereditary transthyretin amyloidosis with polyneuropathy: final analysis of the HELIOS-A randomized treatment extension

Figshare2026-06-15

Long-term efficacy and safety of vutrisiran in hereditary transthyretin amyloidosis with polyneuropathy: final analysis of the HELIOS-A randomized treatment extension

Amyloid2026-06-15

Vutrisiran-Mediated Knockdown of Transthyretin in Patients with ATTR Amyloidosis.

2026-05-27

Efficacy and Safety of Vutrisiran in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: Analysis of the East Asian Subpopulation from HELIOS-A.

2026-03-04

Worsening of Heart Failure in Outpatients With Transthyretin Amyloidosis and Cardiomyopathy in the APOLLO-B Trial.

2025-01-225 citations

Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy

JAMA Neurology2025-01-1317 citations

Patisiran Treatment in the Brazilian Subpopulation of the Phase 3 APOLLO-B Study in Transthyretin Amyloidosis with Cardiomyopathy: Post Hoc Analysis

Arquivos Brasileiros de Cardiologia2025-01-013 citations

Tratamento com Patisirana na Subpopulação Brasileira do Estudo de Fase 3 APOLLO-B em Amiloidose por Transtirretina com Cardiomiopatia: Análise Post Hoc

Arquivos Brasileiros de Cardiologia2025-01-012 citations

HELIOS-A: 18-month randomized treatment extension analysis of vutrisiran in patients with hereditary transthyretin amyloidosis with polyneuropathy

2025-01-01

Impact of Baseline Polyneuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study (P2-11.015)

Neurology2024-04-09

PRIMARY RESULTS FROM APOLLO–B OPEN–LABEL EXTENSION STUDY OF PATISIRAN IN PATIENTS WITH TRANSTHYRETIN CARDIAC AMYLOIDOSIS

European Heart Journal Supplements2024-04-012 citations

Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study.

2024-03-271 citations

Impact of Baseline Neuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study.

2024-03-211 citations

History of polyneuropathy and musculoskeletal manifestations in patients with transthyretin-mediated amyloidosis with cardiomyopathy in APOLLO-B

Arquivos de Neuro-Psiquiatria2024-01-01

Abstract 13273: APOLLO-B, a Study of Patisiran in Patients With Transthyretin Cardiac Amyloidosis: Primary Long-Term Results From the Open-Label Extension Period

Circulation2023-11-076 citations

Evaluation of disease progression in patients with ATTR amyloidosis with cardiomyopathy following treatment with patisiran: post-hoc analysis of the APOLLO-B study

European Heart Journal2023-11-01

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis

New England Journal of Medicine2023-10-25261 citations

Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation.

2023-08-281 citations

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